FDA nod for Sanofi's Xenpozyme, first disease-specific treatment for ASMD

The US Food and Drug Administration (FDA) has approved French pharma major Sanofi’s (Euronext: SAN) Xenpozyme (olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of the rare disease acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.

Xenpozyme is the first therapy indicated specifically for the treatment of ASMD, also known as Niemann-Pick disease, and is currently the only approved treatment for this disease, and the FDA green light follows European approval in June and in Japan in March.

Bill Sibold, executive vice president, head, Specialty Care at Sanofi, commented: “Sanofi teams have been dedicated to bringing hope to patients living with ASMD and their families. This is a devastating and extremely rare disease that affects both children and adults. The approval of Xenpozyme represents the culmination of bold work done in research and development, and our unwavering commitment to this historically overlooked community.”